Strategies to Manage Costs in Idiopathic Pulmonary Fibrosis

Gary M. Owens, M

Until 2014, the only available treatment for IPF, apart from a lung transplant, was palliative care and management of comorbidities to improve patient quality of life.11 In 2014, the US Food and Drug Administration (FDA) approved pirfenidone and nintedanib for IPF, each at a cost of almost $100,000 per patient per year.1,11 Additionally, treatments such as N-acetylcysteine are known to be used off-label in clinical practice.28 The efficacy of all 3 of these treatments was evaluated based on a systemic review of phase 2 and 3 randomized controlled trials in adults with IPF.28 Based on the 9 studies included in this meta-analysis, pirfenidone and nintedanib demonstrated greater effectiveness in slowing the decline in FVC compared with placebo after 1 year of treatment (pirfenidone vs placebo: difference = 0.12 liter (L), 95% confidence interval [CI], 0.03-0.21 L; nintedanib vs placebo: difference = 0.11 L, 95% CI, 0.00-0.22 L). N-acetylcysteine did not demonstrate any significant efficacy compared with placebo. Furthermore, treatment with pirfenidone demonstrated a lower risk of decline in percent predicted FVC of ≥10% over 1 year (odds ratio [OR],0.58; 95% CI, 0.40-0.88). All-cause mortality was reduced with pirfenidone compared with placebo over 1 year (hazard ratio [HR], 0.52; 95% CI, 0.28-0.92). A survival advantage in comparison to placebo was not seen with nintedanib (HR, 0.70; 95% CI, 0.32-1.55), or N-acetylcysteine (HR, 2.00; 95% CI, 0.46-8.62).28 Treatment with pirfenidone has been suggested to improve life expectancy in patients with IPF compared with best supportive care (BSC).29 A sub-analysis of data from 2 randomized clinical studies (ASCEND and CAPACITY), an open-label extension study (RECAP), and the Inova Fairfax Hospital database calculated the mean life expectancy for patients with IPF being treated with pirfenidone at 8.72 (95% CI, 7.65-10.15) years compared with 6.24 (5.38-7.18) years with BSC. This was an improvement in life expectancy of 2.47 (1.26-4.17) with pirfenidone compared with BSC.29

Both FDA-approved agents have demonstrated a modest effect on slowing decline in lung function in patients with IPF.30,31 In contrast, the PANTHER trial showed that combination therapy with prednisone, azathioprine, and N-acetylcysteine results in harm with increased mortality and increased hospitalizations. However, because the agents are relatively new, there is an absence of consensus on when to initiate treatment and a dearth of long-term health outcomes data to support the steep cost associated with treatment. In the absence of these long-term outcomes data treatment guidelines, it is imperative that healthcare providers take all the factors involved in the management of IPF into account, including the cost of treating declining symptoms and the cost of comorbidities.32

Optimizing patient care requires a thorough understanding of the role of new pharmacologic treatments and how they can change the management of patients and the health-economic assessment of IPF. One recent trial demonstrated that nintedanib slowed the decline in lung function independent of the degree of FVC impairment at baseline. In the study, patients with IPF and preserved lung volume (FVC >90% predicted) had the same rate of FVC decline over the following year and received the same benefit from nintedanib as patients with more impaired lung volume.33 Treatment with nintedanib slowed the annual rate of decline in FVC in patients with IPF regardless of predicted FVC. Low or worsening FVC has been shown to be a risk factor for acute exacerbations, and these patients may be more likely to receive treatment.34 There is evidence that patients with less severe impairment in FVC are not as likely to receive treatment.35 However, there is some rationale to early treatment initiation, regardless of FVC. The first is that early treatment initiation can help preserve lung function, potentially delay disease progression, and potentially increase quality of life. The second is that FVC percent predicted is not an absolute indicator of functional lung tissue. In fact, the presence of emphysema increases FVC by modifying the impact of fibrosis on respiration.33,36 These findings of the nintedanib study support the concept of initiating treatment in patients with IPF with preserved lung volumes at the time of diagnosis, rather than waiting for symptoms of progression.

Optimizing Patient Care

As diagnostic criteria become more refined and accurate and potentially effective therapies emerge, attention should be given to healthcare resource usage and healthcare processes that ensure patient-centered management with sustainable, cost-effective, and quality care. As such, it is important to implement a structured, comprehensive, multidisciplinary management approach for the treatment and management of IPF and its associated comorbidities. This may help limit costs and provide effective and quality healthcare. In the case of a chronic condition such as IPF, this includes:

Structured communication between healthcare professionals and the patient

Initiation of care management programs that incorporate patient counseling and professional support

Importance of Communication

The realization of an IPF diagnosis is daunting, and the complexity of the process leading up to the diagnosis is likely to place significant stress on an already difficult situation. Before a diagnosis, approximately 40% of patients with IPF have already consulted 3 or more medical professionals.37 The rapid disease progression may require that patients make decisions about care management, such as whether to opt for lung transplantation versus medical therapy. Unlike cancer or diabetes, educational support for IPF is lacking, and patients are often completely dependent on their healthcare team for support, advice, and disease education. As such, effective communication between the healthcare team and the patient is critical for patient support and effective treatment of this complex disease. This communication is greatly improved when the healthcare provider not only has a thorough understanding of IPF (including its etiology, associated comorbidities, and treatment options), but also an understanding of how the course of the disease impacts the patient on a day-to-day level. A patient-centered care approach includes informed, activated, participatory patient and family; an accessible, well-organized, responsive healthcare system; and a patient-centered communicative clinician who work together to improve communications that lead to improved health outcomes.37 These factors will ensure that health outcomes are improved through responding to emotions, exchanging information, managing uncertainty, enabling patient self-management, fostering healing relationships, and making decisions together.

Care Management Programs

Care management programs are patient centered and are designed to improve the health outcomes and reduce cost due to disease-related complications; this is accomplished through coordinated healthcare interventions and communications for patients with a specific medical condition.38,39 These programs do so by focusing on a target population and select factors that contribute to decreased functional status in patients with chronic diseases.40 Most programs center around the idea that patient education is critical to self-management and overall treatment success.39 Overall, targeted care management programs for people with rare chronic diseases support a partnership between healthcare professionals and patients, and develop a plan of care that focuses on prevention of exacerbations and complications. The plan aims at empowering the patient and includes39:

Population identification processes based on demographic characteristics and healthcare usage

Evidence-based practice guidelines to ensure consistency in diagnosis and treatment

Collaborative practice models using a multidisciplinary team that includes healthcare professionals and support-service providers to educate patients on disease management

Patient education, goal setting, and self-management support through patient counseling. Patient education is focused on prevention of exacerbations and the importance of treatment adherence, and uses behavior modification programs and supplemental services, including home visits, counseling, and appointment reminders

Process and outcomes measurement using patient satisfaction, and health and economic outcomes to evaluate the success or failure of a plan. Outcomes monitored include drug use and treatment-related adverse events to maximize therapeutic efficacy and patient outcomes while minimizing drug-related adverse events and cost

Through patient education, care management programs can also help to facilitate preventive care, which is critically important in the management of chronic conditions including IPF. Examples of preventive care strategies include support for smoking cessation and incentives for yearly influenza vaccination and 5-yearly pneumococcal vaccination. Another example of how a care management program can help patients overcome hurdles is seen in a single center observational study of 40 patients with IPF.41 In an attempt to understand low treatment adherence, the study evaluated the before and after effects of the following patient counseling measures with regard to pirfenidone: (1) slow dose titration to a target regimen and the use of prokinetic agents to lessen gastrointestinal-related intolerance; and (2) the use of sunscreen, sunlight avoidance, and dose interruption and reintroduction to minimize skin-related adverse events. Before the counseling measures, 15% of patients discontinued treatment within the first 6 months and more than half experienced drug-related adverse reactions. Nearly 1 year later that number dropped to zero after clinician-initiated interventions were introduced.41 The study demonstrated that improved adherence and compliance can be achieved by specialist nurse and clinician review, support, and education of the patient. Other common strategies used by care management programs to improve medication adherence include proactively and retroactively monitoring refill rates and educating patients and/or caregivers about drug administration and handling, adverse effects, and the potential for drug interactions.42 Considering that pirfenidone and nintedanib are metabolized by enzymes of the cytochrome P-450 system, screening for drug interactions and communicating this information to patients has the potential to reduce adverse drug-related events and improve patient quality of life.